Work

485. Long-Term In Vivo Engraftment and Transgene Expression from CD34+ Human Hematopoietic Stem Cells Transduced with Pseudotyped rAAV

Year: 2006

Type: article

Abstract: The search for the ideal vector for stem cell gene therapy continues as recognized problems remain with all existing viral vectors. The recent identification of over 100 new serotypes of AAV significantly expands the repertoire of available gene transfer vectors. Additionally, the capacity to pseudotype rAAV2 genomes in novel capsids facilitates evaluation. We and others have analyzed rAAV2-mediated gene transfer into hematopoietic stem cells (HSC). However, despite efficient gene-marking of primitive HSCs, there are identified limitations to transgene expression especially at early time points.

Source: Molecular Therapy

DOI: 10.1016/j.ymthe.2006.08.555

Authors: Sarah Hardy, Margaret Heerwagen, Lane Bilbrey, Kamehameha K. Wong, Saswati Chatterjee

Institutions: City Of Hope National Medical Center, Howard Hughes Medical Institute

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,500